Medications for cystic fibrosis can help control the mucus in the lungs and keep the lungs’ normal functioning as much as possible.
What is Cystic Fibrosis?
Cystic fibrosis is a genetic disease known to affect the cells that make mucus, digestive fluids, and sweat. Usually, these fluids are thin and very slippery to keep the body's systems run smoothly. However, if a person has cystic fibrosis, these secretions become thick and sticky leading to a blockage of the ducts and tubes present throughout the body. Gradually, as time passes, this mucus also builds up inside the airways, making it difficult for the person to breathe. The mucus buildup starts trapping germs, which then leads to infections. In some cases, it can lead to severe lung disorders such as cysts and fibrosis.
Cystic fibrosis currently has no cure but there are various medications available that can help control the symptoms. Medications can help ease the symptoms if they are taken on a timely basis. Moreover, medications are taken to prevent any kind of complications related to the disease. It would make the life of people with cystic fibrosis better by carrying on with their normal routine activities.
Below are some of the medications used for cystic fibrosis. These medications can be inhaled, swallowed, or injected.
- Antibiotics: Antibiotic drugs are used to prevent and treat infections caused by the disease.
- Mucolytic Agents: Medicines such as hypertonic saline, dornase alfa, and mannitol dry powder for thinning the sticky mucus that has blocked the airways.
- Bronchodilators: These drugs are used to widen the airways and allow easier breathing.
- Steroids: Medications that contain steroid are usually prescribed for the treatment of nasal polyps, which are small growths inside the nose.
Mucus Secretion in Cystic Fibrosis
Mucus is very important in our body for maintaining a pair of healthy lungs. Any inhaled particles such as viruses or bacteria can get into the trapped mucus and cilia, which are tiny hair-like structures. Cilia are projections on the surface of the airway cells that help sweep out the mucus from the airways.
The mucus proteins that fail to achieve the correct form of structure lead to the airways and become thick and sticky in patients who are suffering from cystic fibrosis. The lack of water is simply the reason why mucus proteins fail to take up their normal shape or structure.
There are two ways to address the problem related to the mucus buildup. The first possibility could be to increase the content of water atop the cell layer, which is lining the lungs. Secondly, take certain drugs that can help break the mucus proteins into certain smaller pieces when water levels are lower than usual. The mucus is made up of a range of molecules. When there is not enough water in the body, the concentration of proteins and other molecules of mucus rise.
1. Acetylcysteine (Mucomyst)
Mucomyst is a brand of acetylcysteine and is a mucolytic solution for inhalation. It helps to thin down and loosen up mucus, which is present in the airways due to cystic fibrosis. Acetylcysteine reacts to certain chemicals in the mucus, making it less sticky and easier to cough up. This medication would help clear the airways in the lungs letting individuals with cystic fibrosis breathe easier.
To inhale this solution, you would need to use a machine like a nebulizer, which would turn the solution into a mist for inhalation. Acetylcysteine can also be taken by mouth to prevent liver damage from acetaminophen overdose. If one is using acetylcysteine to treat a lung problem due to cystic fibrosis, follow the doctor's advice on how to use the solution. The doctor or nurse can guide you on the proper way of using this medicine and make sure to learn all the instructions properly.
While inhaling the medicine, you may notice a slight odor of the medication. However, this would go away quickly after a period of use. If one is using a face mask for breathing in the medication, then there would be some form of stickiness on the face after use. Once you are done with the procedure, remove the mask and wash your face thoroughly with water.
Do not mix acetylcysteine with any other type of inhaled solutions unless specifically directed by the doctor. If one is taking this medication orally, then follow the doctor's instruction. The solution is usually mixed with another form of liquid to decrease the chances of nausea and vomiting. The medication should be taken within an hour of mixing. The dosage for each individual would be different. The dosage would be according to the patient's overall health status and the severity of the patient's medical condition for which the treatment is carried out.
Acetylcysteine also comes along with certain side effects. Vomiting and nausea are the most common side effects of this medicine. Other side effects include a runny nose, painful mouth sores, fever, drowsiness, and wheezing. Such side effects usually occur in patients who inhale the medicine. Inform the doctor immediately if any of these side effects tend to worsen.
The serious side effects of acetylcysteine are bronchospasm, wheezing, chest pain or tightness, and having trouble while breathing. Allergic reactions to this drug are a rare occurrence. However, inform the doctor if you observe the following signs and symptoms after taking the medication:
- Swelling of the face, tongue, lips or throat
- Severe dizziness
- Irregular breathing
The dosage of the drug and the method on how to take it would depend upon the following factors:
- Age of the patient
- The condition being treated
- The severity of the condition
- The presence of other medical conditions
- Reaction to the first dose
Before starting off with this medicine, inform the doctor if you are allergic to any of the components mentioned in the medicine. This product contains certain inactive ingredients, which can lead to signs of allergic reactions or other problems. Before taking this medicine, inform the doctor if you have asthma or stomach ulcers. During pregnancy, this medicine should be exclusively used only if required and upon consultation with the doctor.
- Adult Dosage: You can use a nebulizer face mask, a mouth piece, or tracheostomy tube. The dose recommended for patients above the age of 18 years old is 3-5 mL of the 20 percent solution or 6-10 mL of the 10 percent solution. The solution should be inhaled for 3-4 times a day.
- Child Dosage: The dose recommended for children under the age of 18 is 3-5 mL of the 20 percent solution or it can be 6-10 mL of the 10 percent solution. This should be carried out at least 3-4 times within 24 hours. The doses should be given in every 2-6 hours.
2. Dornase Alfa (DNase or Pulmozyme)
This medication is prescribed for patients who are suffering from lung infections. Dornase alfa reduces lung infections and also helps improve the proper functioning of the lungs, especially those suffering from cystic fibrosis. This medicine is an enzyme and it works by breaking down the thick mucus secretions that block the airways. It allows a better and free flow of air and prevents any buildup of bacteria that can often lead to infections.
Dornase Alfa is available in the form of a solution, which needs to be inhaled through the mouth. It can be taken once or twice a day. Ensure to follow the directions given by the doctor. You can also check the label of the medicine for instructions on how to use the medicine.
Avoid using more or less of the content solution since it can lead to some other side effects. It is important to note that this medicine is not a cure for cystic fibrosis. It only helps reduce the symptoms and clear the air passage. This medicine can be continuously taken even if one finds relief from the symptoms. Do not stop using this medicine unless the doctor has said so or else it can lead to withdrawal symptoms.
Before starting the medication, inform the doctor if you are allergic to any of the contents present in the medicine. Inform the doctor about any drugs being taken whether it is prescribed or non-prescribed. Also, inform the doctor if you are pregnant, planning to get pregnant, or nursing a baby.
Dornase alfa also has side effects, but they are usually mild. Its side effects include a sore throat, hoarseness in the voice, eye irritation, changes in the voice, and skin rashes. Inform the doctor if any of the symptoms tend to get worse. The serious side effects of the drug may include pain or tightness in the chest and difficulty in breathing. Do not mix dornase alfa with other solutions for inhalation. The device used for inhalation should be properly cleaned and maintained. Moreover, this medication should not be used by anyone else.
3. Hypertonic Salt
It is a sterile form of saline solution in different concentrations. Hypertonic salt works by increasing the amount of sodium in the airways. Salt is known to attract water into the airways. This would help thin the mucus, thereby making it easier to cough up.
Studies have revealed that inhaling hypertonic salt solutions at least twice a day can help individuals suffering from cystic fibrosis. Inhaling the solution can also help reduce different types of lung infections. Those who are above the age of 6 might be able to inhale hypertonic salt solutions. However, before one starts taking it, consult the doctor about it and ask if it is the right medication for you. You can use a nebulizer to inhale the hypertonic solution twice a day. The doctor can guide you on the correct procedure for taking in the medicine.
To make sure that the saline solution contains the correct amount of salt, it is always recommended to use hypertonic salt solutions. Do not take other types of medications along with hypertonic salt. Unlike other medications, hypertonic salt does not stay in the system for too long. Thus, its mucus thinning effect is very low. For this reason, it is quite important to perform an airway clearance technique right after taking dornase alfa. This should be done while the mucus is thin and can be easily removed from the airways by coughing up. Certain side effects of the medicine are sore throat, pain or tightness in the chest, and persistent coughing.